Strategic infection prevention after genetically modified hematopoietic stem cell therapies: recommendations from the International Society for Cell & Gene Therapy Stem Cell Engineering Committee

Tami D John; Gabriela Maron; Allistair Abraham; Alice Bertaina; Senthil Velan Bhoopalan; Alan Bidgoli; Carmem Bonfim; Zane Coleman; Amy DeZern; Jingjing Li; Chrystal Louis; Joseph Oved; Mara Pavel-Dinu; Duncan Purtill; Annalisa Ruggeri; Athena Russell; Robert Wynn; Jaap Jan Boelens; Susan Prockop; Akshay Sharma

doi:10.1016/j.jcyt.2024.02.005

Strategic infection prevention after genetically modified hematopoietic stem cell therapies: recommendations from the International Society for Cell & Gene Therapy Stem Cell Engineering Committee

Cytotherapy. 2024 Jul;26(7):660-671. doi: 10.1016/j.jcyt.2024.02.005. Epub 2024 Feb 20.

Authors

Tami D John¹, Gabriela Maron², Allistair Abraham³, Alice Bertaina¹, Senthil Velan Bhoopalan⁴, Alan Bidgoli⁵, Carmem Bonfim⁶, Zane Coleman², Amy DeZern⁷, Jingjing Li⁸, Chrystal Louis⁹, Joseph Oved¹⁰, Mara Pavel-Dinu¹, Duncan Purtill¹¹, Annalisa Ruggeri¹², Athena Russell¹³, Robert Wynn¹⁴, Jaap Jan Boelens¹⁰, Susan Prockop¹⁵, Akshay Sharma¹⁶

Affiliations

¹ Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University, Stanford, California, USA.
² Department of Infectious Diseases, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
³ Center for Cancer and Immunology Research, CETI, Children's National Hospital, Washington, District of Columbia, USA.
⁴ Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
⁵ Division of Blood and Marrow Transplantation, Children's Healthcare of Atlanta, Aflac Blood and Cancer Disorders Center, Emory University, Atlanta, Georgia, USA.
⁶ Pediatric Blood and Marrow Transplantation Division and Pelé Pequeno Príncipe Research Institute, Hospital Pequeno Príncipe, Curitiba, Brazil.
⁷ Bone Marrow Failure and MDS Program, John Hopkins Medicine, Baltimore, Maryland, USA.
⁸ Graduate School of Biomedical Engineering, University of New South Wales, Sydney, New South Wales, Australia.
⁹ Zentalis Pharmaceuticals, New York, New York, USA.
¹⁰ Stem Cell Transplantation and Cellular Therapies Service, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
¹¹ Department of Haematology, Fiona Stanley Hospital, Perth, Western Australia, Australia.
¹² IRCCS Ospedale San Raffaele, Segrate, Milan, Italy.
¹³ Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
¹⁴ Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK.
¹⁵ Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.
¹⁶ Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA. Electronic address: [email protected].

PMID: 38483362
PMCID: PMC11213676 (available on 2025-07-01)
DOI: 10.1016/j.jcyt.2024.02.005

Abstract

There is lack of guidance for immune monitoring and infection prevention after administration of ex vivo genetically modified hematopoietic stem cell therapies (GMHSCT). We reviewed current infection prevention practices as reported by providers experienced with GMHSCTs across North America and Europe, and assessed potential immunologic compromise associated with the therapeutic process of GMHSCTs described to date. Based on these assessments, and with consensus from members of the International Society for Cell & Gene Therapy (ISCT) Stem Cell Engineering Committee, we propose risk-adapted recommendations for immune monitoring, infection surveillance and prophylaxis, and revaccination after receipt of GMHSCTs. Disease-specific and GMHSCT-specific considerations should guide decision making for each therapy.

Keywords: genetically modified hematopoietic stem cell therapies; immune reconstitution; prophylaxis; vaccination.

Publication types

Review

MeSH terms

Cell- and Tissue-Based Therapy / methods
Genetic Therapy* / methods
Hematopoietic Stem Cell Transplantation* / adverse effects
Hematopoietic Stem Cell Transplantation* / methods
Hematopoietic Stem Cells / cytology
Humans
Infections / etiology
Infections / therapy

Abstract

Publication types

MeSH terms

Grants and funding